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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
i
Other names: Platelet Derived Growth Factor Receptor Alpha, Platelet-Derived Growth Factor Receptor Alpha Polypeptide, Alpha-Type Platelet-Derived Growth Factor Receptor, CD140 Antigen-Like Family Member A, CD140a Antigen, PDGF-R-Alpha, PDGFR-2, PDGFR2, Alpha Platelet-Derived Growth Factor Receptor, Platelet-Derived Growth Factor Alpha Receptor, PDGFR-Alpha, RHEPDGFRA, CD140A
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News alerts, weekly reports and conference planners
6d
Somatic mutational profiling and clinical impact of driver genes in Latin-Iberian medulloblastomas: Towards precision medicine. (PubMed, Neuropathology)
Latin-Iberian medulloblastomas, particularly the MBSHH, exhibit higher mutation frequencies than other populations. We corroborate the TP53 mutation status as an important prognostic factor, while PIK3CA and KIT are potential therapeutic targets.
Journal
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EGFR (Epidermal growth factor receptor) • HER-2 (Human epidermal growth factor receptor 2) • TP53 (Tumor protein P53) • PIK3CA (Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha) • NRAS (Neuroblastoma RAS viral oncogene homolog) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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TP53 mutation • PIK3CA mutation
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TruSight Tumor 15 Assay
7d
Primary Cardiac Intimal Sarcoma: Multi-Layered Strategy and Core Role of MDM2 Amplification/Co-Amplification and MDM2 Immunostaining. (PubMed, Diagnostics (Basel))
To summarize, MDM2 amplification and co-amplification, for example, with MDM4, CDK4, HMGA3, CCND3, PDGFRA, TERT, KIT, CCND3, and HDAC9, might improve the diagnosis of PCIS in addition to MDM2 immunostaining since 10-20% of these tumours are MDM2-negative. Further studies are necessary to highlight MDM2 applicability as a prognostic factor and as an element to be taken into account amid multi-layered management in an otherwise very aggressive malignancy.
Review • Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • MDM2 (E3 ubiquitin protein ligase) • CDK4 (Cyclin-dependent kinase 4) • MDM4 (The mouse double minute 4) • CCND3 (Cyclin D3) • HDAC9 (Histone Deacetylase 9)
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MDM2 amplification
7d
Tobacco Smoke Condensate Induces Morphologic Changes in Human Papillomavirus-Positive Cervical Epithelial Cells Consistent with Epithelial to Mesenchymal Transition (EMT) with Activation of Receptor Tyrosine Kinases and Regulation of TGFB. (PubMed, Int J Mol Sci)
Our study demonstrated that CSC induces EMT in ectocervical cells with the upregulation of EMT-related genes, expression of protein biomarkers, and activation of RTKs that regulate TGFB expression, and other EMT-related genes. Understanding the molecular pathways and environmental factors that initiate EMT in ectocervical cells will help delineate molecular targets for intervention and define the role of EMT in the initiation and progression of cervical intraepithelial neoplasia and CC.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FGFR (Fibroblast Growth Factor Receptor) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • CDH1 (Cadherin 1) • VIM (Vimentin) • DDR2 (Discoidin domain receptor 2) • SNAI2 (Snail Family Transcriptional Repressor 2) • SMAD2 (SMAD Family Member 2)
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VIM expression
7d
Utility of Clinical Next Generation Sequencing Tests in KIT/PDGFRA/SDH Wild-Type Gastrointestinal Stromal Tumors. (PubMed, Cancers (Basel))
Compared to KIT/PDGFRA-mutant GIST, limited benefit was observed with imatinib in triple-negative GIST. In depth molecular profiling can be helpful in identifying driver mutations and guiding therapy.
Journal • Next-generation sequencing • Stroma
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BRAF (B-raf proto-oncogene) • TP53 (Tumor protein P53) • PTEN (Phosphatase and tensin homolog) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • NTRK3 (Neurotrophic tyrosine kinase, receptor, type 3) • FGFR1 (Fibroblast growth factor receptor 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • ETV6 (ETS Variant Transcription Factor 6) • CHEK2 (Checkpoint kinase 2) • FANCA (FA Complementation Group A) • SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • AURKA (Aurora kinase A) • SDHC (Succinate Dehydrogenase Complex Subunit C) • SDHD (Succinate Dehydrogenase Complex Subunit D) • SDHA (Succinate Dehydrogenase Complex Flavoprotein Subunit A)
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TP53 mutation • BRAF V600E • BRAF V600 • NTRK3 fusion • PTEN deletion • PTEN mutation • NF1 mutation • ETV6-NTRK3 fusion • CHEK2 mutation • PDGFRA mutation • FANCA mutation • FGFR1 fusion • FANCA deletion • PDGFR wild-type
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imatinib
8d
Inducible CXCL12/CXCR4-dependent extramedullary hematopoietic niches in the adrenal gland. (PubMed, Blood)
Mirroring our findings in mice, we found reticular CXCL12+ cells co-expressing master niche-regulator FOXC1 in primary samples from human adrenal myelolipomas, a benign tumor composed of adipose and hematopoietic tissue. Our findings reignite long-standing questions regarding hormonal regulation of hematopoiesis and provide a novel model to facilitate the study of adult-specific inducible hematopoietic niches which may pave the way to therapeutic applications.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CXCR4 (Chemokine (C-X-C motif) receptor 4) • CXCL12 (C-X-C Motif Chemokine Ligand 12) • FOXC1 (Forkhead Box C1)
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CXCL12 expression
9d
Monitoring advanced gastrointestinal stromal tumor with circulating tumor DNA. (PubMed, Curr Opin Oncol)
The dynamic field of ctDNA technologies is rapidly evolving and holds significant promise for research. Several trials have successfully validated the clinical utility of ctDNA in metastatic GIST, laying the foundations for its prospective integration into the routine clinical management of GIST patients.
Clinical • Journal • Circulating tumor DNA • Stroma • Metastases
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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PDGFRA mutation
9d
The Effect of Therapy Regimen on Antitumor Efficacy of the Nanosomal Doxorubicin against Rat Glioblastoma 101.8. (PubMed, Bull Exp Biol Med)
Early start of treatment prolonged the survival time and increased tumor growth inhibition by 141.4 and 95.7%, respectively, in comparison with the untreated group; these differences were not observed in the group with late start of therapy. The results indicate that the time of initiation of therapy is a critical parameter affecting the antitumor efficacy of DOX-PLGA.
Preclinical • Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • MELK (Maternal Embryonic Leucine Zipper Kinase)
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doxorubicin hydrochloride
11d
Myxoid Inflammatory Myofibroblastic Sarcoma: Clinicopathologic Analysis of 25 Cases of a Distinctive Sarcoma With Deceptively Bland Morphology and Aggressive Clinical Behavior. (PubMed, Am J Surg Pathol)
The patient whose tumor harbored SEC31A::PDGFRA was treated after multiple relapses with imatinib and sunitinib therapy, with progression-free periods of 5 and 2 years, respectively. Despite its bland appearance, myxoid inflammatory myofibroblastic sarcoma harbors a significant risk for disseminated disease, particularly when it occurs in the peritoneum. Targeted therapy could be considered for patients with disseminated disease.
Journal
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KRAS (KRAS proto-oncogene GTPase) • ALK (Anaplastic lymphoma kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • TNFRSF8 (TNF Receptor Superfamily Member 8) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • JAK1 (Janus Kinase 1) • SEC31A (SEC31 Homolog A COPII Coat Complex Component)
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KRAS mutation • ALK rearrangement • KRAS G12V • TNFRSF8 expression • KRAS G12 • KRAS Q61H
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imatinib • sunitinib
12d
Development of 42 marker panel for in-depth study of cancer associated fibroblast niches in breast cancer using imaging mass cytometry. (PubMed, Front Immunol)
The final panel contained 6 CAF-associated markers (aSMA, FAP, PDGFRa, PDGFRb, YAP1, pSMAD2). Breast cancer tissues (4 cases of luminal, 5 cases of triple negative breast cancer) and a modified CELESTA pipeline were used to demonstrate the utility of our IMC panel for detailed profiling of different CAF, immune and cancer cell phenotypes.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • YAP1 (Yes associated protein 1)
18d
Marked eosinophilic atypia in a patient with asthma and myeloid/lymphoid neoplasm with FIP1L1::PDGFRA fusion. (PubMed, Br J Haematol)
No abstract available
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FIP1L1 (Factor Interacting With PAPOLA And CPSF1)
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FIP1L1-PDGFRA fusion • PDGFRA fusion
20d
Brefeldin A and M-COPA block the export of RTKs from the endoplasmic reticulum via simultaneous inactivation of ARF1, ARF4, and ARF5. (PubMed, J Biol Chem)
Inhibitors of protein export from the endoplasmic reticulum (ER), such as brefeldin A (BFA) and 2-methylcoprophilinamide (M-COPA), can suppress the activation of mutant RTKs in cancer cells, suggesting that RTK mutants cannot initiate signaling in the ER...Consistent with these results, in vitro pulldown assays showed that BFA/M-COPA blocked the function of ARF1, ARF4, and ARF5. Taken together, these results suggest that BFA/M-COPA targets at least ARF1, ARF4, and ARF5; in other words, RTKs require the simultaneous activation of ARF1, ARF4, and ARF5 for their ER export.
Journal
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EGFR (Epidermal growth factor receptor) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
22d
Morphological and Molecular Biological Characteristics of Experimental Rat Glioblastoma Tissue Strains Induced by Different Carcinogenic Chemicals. (PubMed, Biomedicines)
GBM 101.8 is a reliable model for further investigation due to its similarity to high-grade human GBMs, while GBM 11-9-2 and GBM 14-4-5 correspond to Grade 2-3 gliomas.
Preclinical • Journal
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TP53 (Tumor protein P53) • PTEN (Phosphatase and tensin homolog) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • MGMT (6-O-methylguanine-DNA methyltransferase) • ABCB1 (ATP Binding Cassette Subfamily B Member 1) • HIF1A (Hypoxia inducible factor 1, alpha subunit) • SOX2
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ABCB1 overexpression • HIF1A expression • VEGFA expression • MGMT expression • SOX2 expression
22d
Fusion of Platelet Derived Growth Factor Receptor Alpha (PDGFRA) With Ubiquitin Specific Peptidase 8 (USP8) in a Calcified Chondroid Mesenchymal Neoplasm Harboring t(4;15)(q12;q21) as a Sole Aberration. (PubMed, Cancer Genomics Proteomics)
We report, for the first time, a calcified chondroid mesenchymal neoplasm carrying a balanced t(4;15)(q12;q21) chromosomal translocation, resulting in the generation of both PDGFRA::USP8 and USP8::PDGFRA chimeras. The PDGFRA::USP8 protein is located on the cell membrane and functions as a chimeric ubiquitinyl hydrolase, activated by PDGFs. Conversely, USP8::PDGFRA is a nuclear protein regulating metabolic processes.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FN1 (Fibronectin 1) • NECTIN1 (Nectin Cell Adhesion Molecule 1)
22d
Blockade of Discoidin Domain Receptor Signaling with Sitravatinib Reveals DDR2 as a Mediator of Neuroblastoma Pathogenesis and Metastasis. (PubMed, Mol Cancer Ther)
Analysis of single cell sequencing data demonstrated that DDR2 is restricted to mesenchymal-type tumor subpopulations and is enriched in Schwann Cell Precursor (SCP) subpopulations found in high-risk disease. These data define an unsuspected role for sitravatinib as a therapeutic agent in neuroblastoma and reveal a novel function for DDR2 as a driver of tumor growth and metastasis.
Journal
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ALK (Anaplastic lymphoma kinase) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • DDR2 (Discoidin domain receptor 2)
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sitravatinib (MGCD516)
24d
A Study Comparing the Combination of Dasatinib and Chemotherapy Treatment With or Without Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or Philadelphia Chromosome-Like (Ph-Like) ABL-Class B-Cell Acute Lymphoblastic Leukemia (B-ALL) (clinicaltrials.gov)
P3; Initiation date: Apr 2024 --> Jul 2024
Trial initiation date
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ABL1 (ABL proto-oncogene 1) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CD19 (CD19 Molecule) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • CSF1R (Colony stimulating factor 1 receptor)
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CD19 expression
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TruSight RNA Pan-Cancer Panel
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dasatinib • imatinib • cytarabine • doxorubicin hydrochloride • cyclophosphamide • Blincyto (blinatumomab) • methotrexate • vincristine • daunorubicin • leucovorin calcium • Oncaspar liquid (pegaspargase) • mercaptopurine • Asparlas (calaspargase pegol-mknl) • thioguanine • Starasid (cytarabine ocfosfate)
26d
Cancer-associated fibroblast activation predicts progression, metastasis, and prognosis of cutaneous squamous cell carcinoma. (PubMed, Int J Cancer)
Multimarker combination defined CAF subset, PDGFRα-/PDGFRβ+/FAP+, was associated with invasion and metastasis, and independently predicted poor disease-specific survival. These results identify high PDGFRβ expression alone and multimarker combination PDGFRα-/PDGFRβ+/FAP+ by CAFs as potential biomarkers for risk of metastasis and poor prognosis.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • SPARC (Secreted Protein Acidic And Cysteine Rich) • POSTN (Periostin)
26d
Molecular diversity in isocitrate dehydrogenase-wild-type glioblastoma. (PubMed, Brain Commun)
A successful glioblastoma management demands biomarker identification, combination therapies and a nuanced approach considering intratumoural variability. These advancements herald a transformative era in glioblastoma comprehension and treatment.
Review • Journal
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EGFR (Epidermal growth factor receptor) • PTEN (Phosphatase and tensin homolog) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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EGFR expression • IDH wild-type • NF1 overexpression
1m
Autophagy cooperates with PDGFRA to support oncogenic growth signaling. (PubMed, Autophagy)
Activation of downstream signaling through Pten mutation overrides the need for autophagy during tumor development suggesting a genotype-specific role for autophagy during tumorigenesis. Altogether, our findings provide a novel mechanism through which autophagy can support tumor growth.
Journal
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PTEN (Phosphatase and tensin homolog) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • SQSTM1 (Sequestosome 1) • PDGFA (Platelet Derived Growth Factor Subunit A)
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PTEN mutation
1m
ETV6::NTRK3 Fusion-Positive Wild-Type Gastrointestinal Stromal Tumor (GIST) with Abundant Lymphoid Infiltration (TILs and Tertiary Lymphoid Structures): A Report on a New Case with Therapeutic Implications and a Literature Review. (PubMed, Int J Mol Sci)
The follow-up CT scan revealed peritoneal nodules suggestive of peritoneal dissemination, and Entrectinib (a TRK inhibitor) was administered...The present case may offer new insights into the potential introduction of TRK inhibitors as treatments for GISTs with NTRK fusions. Additionally, the presence of abundant lymphoid infiltration in the present case may prompt further research into immunotherapy as a possible additional therapeutic option.
Clinical • Observational data • Retrospective data • Review • Journal • IO biomarker • Stroma
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KIT (KIT proto-oncogene, receptor tyrosine kinase) • NTRK3 (Neurotrophic tyrosine kinase, receptor, type 3) • RB1 (RB Transcriptional Corepressor 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • ETV6 (ETS Variant Transcription Factor 6) • SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • TFG (Trafficking From ER To Golgi Regulator) • NTRK (Neurotrophic receptor tyrosine kinase) • ANO1 (Anoctamin 1)
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NTRK3 fusion • RB1 mutation • PDGFRA mutation • NTRK3 positive • NTRK fusion
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Rozlytrek (entrectinib)
1m
Pediatric-type high-grade gliomas with PDGFRA amplification in adult patients with Li-Fraumeni syndrome: clinical and molecular characterization of three cases. (PubMed, Acta Neuropathol Commun)
These data suggest that high-grade gliomas developed in adult patients with LFS may be involved in pHGG H3-/IDH-wt. PDGFRA and homozygous alterations in TP53 may play pivotal roles in the development of this type of glioma in adult patients with LFS.
Journal
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EGFR (Epidermal growth factor receptor) • TP53 (Tumor protein P53) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • TERT (Telomerase Reverse Transcriptase)
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TP53 mutation • EGFR mutation • EGFR amplification • TERT mutation • IDH wild-type • TERT promoter mutation • PDGFR wild-type
1m
Bone destruction was the initial symptom in myeloid/lymphoid neoplasms associated with eosinophilia and rearrangements of PDGFRα: a case report (PubMed, Zhonghua Xue Ye Xue Za Zhi)
No abstract available
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
1m
Journal • Stroma
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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sorafenib
1m
A First-in-human (FIH) Study of IDRX-42 in Participants With Metastatic and/or Unresectable Gastrointestinal Stromal Tumors (GIST) [Study ID: StrateGIST 1] (clinicaltrials.gov)
P1, N=240, Recruiting, IDRx, Inc. | N=143 --> 240
Enrollment change • Stroma • Metastases
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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KIT mutation • PDGFRA mutation • PDGFRA exon 18 mutation
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sunitinib • IDRX-42
1m
Quantifying intra-tumoral genetic heterogeneity of glioblastoma toward precision medicine using MRI and a data-inclusive machine learning algorithm. (PubMed, PLoS One)
This study demonstrated the feasibility of using MRI and WSO-SVM to enable non-invasive prediction of intra-tumoral regional genetic alteration for each GBM patient, which can inform future adaptive therapies for individualized oncology.
Journal • Machine learning
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EGFR (Epidermal growth factor receptor) • PTEN (Phosphatase and tensin homolog) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
1m
Mesenchymal-specific Alms1 knockout in mice recapitulates metabolic features of Alström Syndrome. (PubMed, Mol Metab)
Mesenchymal deletion of Alms1 recapitulates metabolic features of AS, including fatty liver. This confirms a key role for Alms1 in the adipose lineage, where its loss is sufficient to cause systemic metabolic effects and damage to remote organs. Hyperphagia in females may depend on Alms1 deficiency in oligodendrocyte precursor cells rather than neurones. AS should be regarded as a forme fruste of lipodystrophy.
Preclinical • Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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FOLR1 expression • PDGFRA expression
1m
Single-cell approaches define two groups of mammalian oligodendrocyte precursor cells and their evolution over developmental time. (PubMed, Stem Cell Reports)
When adult oligodendrogenesis is enhanced during experimentally induced remyelination, adult OPCs do not reacquire a developmental open chromatin state, and the oligodendrogenesis trajectory is distinct from that seen neonatally. These data suggest that there are two OPC groups subserving distinct postnatal functions and that neonatal and adult OPC-mediated oligodendrogenesis are fundamentally different.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
1m
The relationships of platelet-derived growth factor, microvascular proliferation, and tumor cell proliferation in canine high-grade oligodendrogliomas: Immunohistochemistry of 45 tumors and an AFOB-01 xenograft mouse model. (PubMed, Vet Pathol)
Crenolanib (a PDGFR inhibitor) inhibited AOFB-01 cell proliferation...Therefore, PDGF-A produced by microvascular proliferations and tumor cells may promote the proliferation of PDGFR-α-expressing tumor cells in canine HGOG. PDGFR-α signaling has potential as a therapeutic target.
Preclinical • Journal • Tumor cell
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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crenolanib (ARO-002)
2ms
Imatinib TDM in GIST (clinicaltrials.gov)
P2, N=28, Recruiting, Reema A. Patel | Not yet recruiting --> Recruiting | Trial primary completion date: Dec 2024 --> Dec 2025
Enrollment open • Trial primary completion date • Stroma
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KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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KIT mutation • PDGFRA D842V • PDGFRA mutation
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imatinib
2ms
Buyang huanwu decoction promotes remyelination via miR-760-3p/GPR17 axis after intracerebral hemorrhage. (PubMed, J Ethnopharmacol)
BYHWD improves remyelination and neurological function in ICH mice by targeting miR-760-3p to inhibit GPR17. This study may shed light on the orchestration of remyelination mechanisms after ICH, thus providing novel insights for developing innovative prescriptions with brain-protective properties.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PCNA (Proliferating cell nuclear antigen) • MIR760 (MicroRNA 760) • OLIG2 (Oligodendrocyte Transcription Factor 2)
2ms
PDGFRA K385 mutants in myxoid glioneuronal tumors promote receptor dimerization and oncogenic signaling. (PubMed, Sci Rep)
Furthermore, we evaluated the sensitivity of these mutants to three FDA-approved tyrosine kinase inhibitors: imatinib, dasatinib, and avapritinib, which effectively suppressed the constitutive activity of the mutant receptors. Interestingly, K385M was reported in a few cases of brain tumors but not in MGNT. Our results provide valuable insights into the molecular mechanism underlying the activation of PDGFRα by the K385I/L mutations, highlighting their potential as actionable targets in the treatment of myxoid glioneuronal tumors.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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PDGFRA mutation
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dasatinib • imatinib • Ayvakit (avapritinib)
2ms
World Health Organization and International Consensus Classification of eosinophilic disorders: 2024 update on diagnosis, risk stratification, and management. (PubMed, Am J Hematol)
Identification of rearranged PDGFRA or PDGFRB is critical because of the exquisite responsiveness of these diseases to imatinib. Pemigatinib was recently approved for patients with relapsed or refractory FGFR1-rearranged neoplasms...Hydroxyurea and interferon-α have demonstrated efficacy as initial treatment and in steroid-refractory cases of HES. Mepolizumab, an interleukin-5 (IL-5) antagonist monoclonal antibody, is approved by the U.S Food and Drug Administration for patients with idiopathic HES. Cytotoxic chemotherapy agents, and hematopoietic stem cell transplantation have been used for aggressive forms of HES and CEL, with outcomes reported for limited numbers of patients. Targeted therapies such as the IL-5 receptor antibody benralizumab, IL-5 monoclonal antibody depemokimab, and various tyrosine kinase inhibitors for MLN-eo-TK, are under active investigation.
Journal
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FGFR1 (Fibroblast growth factor receptor 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • IL5 (Interleukin 5)
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imatinib • Pemazyre (pemigatinib) • hydroxyurea
2ms
Preclinical evaluation of protein synthesis inhibitor omacetaxine in pediatric brainstem gliomas. (PubMed, Neurooncol Adv)
Despite these promising in vitro effects, omacetaxine's efficacy in an orthotopic DIPG model was limited due to inadequate penetration across the blood-brain barrier. As such, further research and advancements are crucial to improve the drug's brain penetration, thus enhancing its overall therapeutic potential.
Preclinical • Journal • PARP Biomarker
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CASP3 (Caspase 3) • ANXA5 (Annexin A5)
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Synribo (omacetaxine mepesuccinate)
2ms
Advancing the Management of Skull Base Chondrosarcomas: A Systematic Review of Targeted Therapies. (PubMed, J Pers Med)
The present review offers a comprehensive analysis of targeted therapeutics for skull base chondrosarcomas, highlighting a complex landscape characterized by a range of treatment approaches and new opportunities for tailored interventions. The combination of results from molecular research and clinical trials emphasizes the necessity for specialized treatment strategies and the complexity of chondrosarcoma biology.
Review • Journal • PD(L)-1 Biomarker • IO biomarker
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • COL2A1 (Collagen Type II Alpha 1 Chain)
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MG132
2ms
Ribonucleic Acid Sequencing Reveals the Upregulation and Resolution of Inflammation and Extracellular Matrix Remodeling in Lidocaine-Treated Human Acute Monocytic Leukemia Cell Line THP-1. (PubMed, Biomedicines)
However, a lidocaine dose lower than 0.5 mM induced a mixed anti-inflammatory profile by significantly upregulating tissue remodeling (GDF15, FGF7, HGF, COL4A3, COL8A2, LAMB2, LAMC2, PDGFRA, and VEGFA) and through the resolution of inflammation (Cpeb4, Socs1, Socs2, Socs3, Dusp1, Tnfaip3, and Gata3) gene cassettes. This study explores the effect of lidocaine on the THP-1 in the M2-like healing phenotype and provides potential applications of lidocaine's therapeutic effectiveness in dental tissue repair.
Preclinical • Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • GDF15 (Growth differentiation factor 15) • TNFAIP3 (TNF Alpha Induced Protein 3) • SOCS1 (Suppressor Of Cytokine Signaling 1) • LAMC2 (Laminin subunit gamma 2) • GATA3 (GATA binding protein 3) • SOCS2 (Suppressor Of Cytokine Signaling 2) • DUSP1 (Dual Specificity Phosphatase 1) • FGF7 (Fibroblast Growth Factor 7) • LAMC1 (Laminin Subunit Gamma 1) • SOCS3 (Suppressor Of Cytokine Signaling 3)
2ms
Population pharmacokinetic-pharmacodynamic modeling of serum biomarkers as predictors of tumor dynamics following lenvatinib treatment in patients with radioiodine-refractory differentiated thyroid cancer (RR-DTC). (PubMed, CPT Pharmacometrics Syst Pharmacol)
Longitudinal tumor measurements over time were reasonably well defined by a tumor growth inhibition Emax model, which in addition to lenvatinib exposure, included model-predicted relative changes from baseline over time for Tie-2 and Ang-2 as having significant association with tumor response. The developed PK/PD models pave the way for dose optimization and potential prediction of clinical response.
PK/PD data • Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • FGF23 (Fibroblast Growth Factor 23)
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Lenvima (lenvatinib)
2ms
Radiation-induced effects on TGF-β and PDGF receptor signaling in cancer-associated fibroblasts. (PubMed, Cancer Rep (Hoboken))
Our data demonstrate that IR by itself is insufficient to induce measurable changes in PDGF or TGF-β receptor expression levels or to induce receptor activation in CAFs. However, in the presence of their respective ligands, exposure to radiation at certain doses appear to interfere with TGF-β receptor signaling.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • TGFB1 (Transforming Growth Factor Beta 1)
2ms
SYNERGY-AI: Artificial Intelligence Based Precision Oncology Clinical Trial Matching and Registry (clinicaltrials.gov)
P=N/A, N=50000, Recruiting, Massive Bio, Inc. | Trial completion date: Jun 2025 --> Jun 2027 | Trial primary completion date: Dec 2024 --> Dec 2026
Trial completion date • Trial primary completion date
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EGFR (Epidermal growth factor receptor) • HER-2 (Human epidermal growth factor receptor 2) • PD-L1 (Programmed death ligand 1) • KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • ER (Estrogen receptor) • ALK (Anaplastic lymphoma kinase) • TP53 (Tumor protein P53) • TMB (Tumor Mutational Burden) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • NRAS (Neuroblastoma RAS viral oncogene homolog) • BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset) • FGFR2 (Fibroblast growth factor receptor 2) • PTEN (Phosphatase and tensin homolog) • ROS1 (Proto-Oncogene Tyrosine-Protein Kinase ROS) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • NTRK1 (Neurotrophic tyrosine kinase, receptor, type 1) • BCL2 (B-cell CLL/lymphoma 2) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NTRK3 (Neurotrophic tyrosine kinase, receptor, type 3) • NTRK2 (Neurotrophic tyrosine kinase, receptor, type 2) • STK11 (Serine/threonine kinase 11) • NPM1 (Nucleophosmin 1) • HRAS (Harvey rat sarcoma viral oncogene homolog) • DNMT3A (DNA methyltransferase 1) • ERBB3 (V-erb-b2 avian erythroblastic leukemia viral oncogene homolog 3) • RB1 (RB Transcriptional Corepressor 1) • CLDN18 (Claudin 18) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • JAK2 (Janus kinase 2) • NRG1 (Neuregulin 1) • POLE (DNA Polymerase Epsilon) • AKT1 (V-akt murine thymoma viral oncogene homolog 1) • PD-1 (Programmed cell death 1) • CCND1 (Cyclin D1) • MCL1 (Myeloid cell leukemia 1) • KDR (Kinase insert domain receptor) • PBRM1 (Polybromo 1) • BAP1 (BRCA1 Associated Protein 1) • VEGFA (Vascular endothelial growth factor A) • BCL6 (B-cell CLL/lymphoma 6) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • PTCH1 (Patched 1) • FGFR4 (Fibroblast growth factor receptor 4) • MSH6 (MutS homolog 6) • CDK4 (Cyclin-dependent kinase 4) • MSH2 (MutS Homolog 2) • CTNNB1 (Catenin (cadherin-associated protein), beta 1) • MAP2K2 (Mitogen-activated protein kinase kinase 2) • FBXW7 (F-Box And WD Repeat Domain Containing 7) • WT1 (WT1 Transcription Factor) • ATRX (ATRX Chromatin Remodeler) • BRCA (Breast cancer early onset) • RAF1 (Raf-1 Proto-Oncogene Serine/Threonine Kinase) • TSC2 (TSC complex subunit 2) • CHEK2 (Checkpoint kinase 2) • PD-L2 (Programmed Cell Death 1 Ligand 2) • ERBB4 (erb-b2 receptor tyrosine kinase 4) • JAK1 (Janus Kinase 1) • FANCA (FA Complementation Group A) • TSC1 (TSC complex subunit 1) • MDM4 (The mouse double minute 4) • POLD1 (DNA Polymerase Delta 1) • CDK6 (Cyclin-dependent kinase 6) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • PARP1 (Poly(ADP-Ribose) Polymerase 1) • AURKA (Aurora kinase A) • JAK3 (Janus Kinase 3) • RICTOR (RPTOR Independent Companion Of MTOR Complex 2) • CHEK1 (Checkpoint kinase 1) • GATA6 (GATA Binding Protein 6) • MSH3 (MutS Homolog 3) • TGFBR2 (Transforming Growth Factor Beta Receptor 2) • GNAS (GNAS Complex Locus) • MYCL (MYCL Proto-Oncogene BHLH Transcription Factor) • AKT2 (V-akt murine thymoma viral oncogene homolog 2) • CCND2 (Cyclin D2) • CCND3 (Cyclin D3) • CSF1R (Colony stimulating factor 1 receptor) • MAP3K1 (Mitogen-Activated Protein Kinase Kinase Kinase 1) • ERCC4 (ERCC Excision Repair 4, Endonuclease Catalytic Subunit) • HDAC1 (Histone Deacetylase 1) • PRDM1 (PR/SET Domain 1) • ZNF217 (Zinc Finger Protein 217) • CDKN1A (Cyclin-dependent kinase inhibitor 1A) • GATA3 (GATA binding protein 3) • PARP2 (Poly(ADP-Ribose) Polymerase 2) • PARP3 (Poly(ADP-Ribose) Polymerase Family Member 3) • SDHA (Succinate Dehydrogenase Complex Flavoprotein Subunit A) • ACVR1B (Activin A Receptor Type 1B) • ZNF703 (Zinc Finger Protein 703)
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HER-2 mutation • BAP1 mutation • AKT1 mutation • FGFR3 fusion • JAK3 mutation
2ms
Advancing Precision Oncology in Hereditary Paraganglioma-Pheochromocytoma Syndromes: Integrated Interpretation and Data Sharing of the Germline and Tumor Genomes. (PubMed, Cancers (Basel))
After processing, 8600 variants were submitted programmatically from the INT2GRATE|HPPGL platform to ClinVar via a custom-made INT2GRATE|HPPGL variant submission schema and an application programming interface (API). This novel integrated variant assessment and data sharing in hereditary cancers aims to improve the clinical assessment of genomic variants and advance precision oncology.
Journal
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • SDHB (Succinate Dehydrogenase Complex Iron Sulfur Subunit B) • SDHA (Succinate Dehydrogenase Complex Flavoprotein Subunit A)
2ms
Aggressive systemic mastocytosis with the co-occurrence of PRKG2::PDGFRB, KAT6A::NCOA2, and RXRA::NOTCH1 fusion transcripts and a heterozygous RUNX1 frameshift mutation. (PubMed, Cancer Genet)
The patient rapidly evolved towards SM-AHN, characterized by the persistence of the PRKG2::PDGFRB chimera, due to the presence of an extra copy of the der(5)t(4;5)(q24;q34) chromosome and an increase in the RUNX1 mutation allelic frequency. The results indicated that the transcriptional landscape and the mutational profile of SM deserve attention to predict the evolution and prognosis of this complex disease, whose classification criteria are still a matter of debate.
Journal
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KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NOTCH1 (Notch 1) • JAK2 (Janus kinase 2) • RUNX1 (RUNX Family Transcription Factor 1) • PDGFRB (Platelet Derived Growth Factor Receptor Beta) • KAT6A (Lysine Acetyltransferase 6A) • NCOA2 (Nuclear Receptor Coactivator 2)
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RUNX1 mutation • KIT D816V • JAK2 V617F
2ms
A landscape of patient-derived cancer-associated fibroblast signals in endometrial cancers. (PubMed, Am J Cancer Res)
We demonstrate that endometrial CAFs can be cultured in an enzymatic-digestion-independent manner, and their signaling landscape can be mapped toward understanding CAF-TME dialogue. Our data will help unearth the functional relevance of endometrial CAFs in the context of clinical outcomes and designing CAF-inclusive therapy in the future.
Journal • PD(L)-1 Biomarker • IO biomarker
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PD-L1 (Programmed death ligand 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CD8 (cluster of differentiation 8) • CXCR4 (Chemokine (C-X-C motif) receptor 4) • HIF1A (Hypoxia inducible factor 1, alpha subunit) • CXCL12 (C-X-C Motif Chemokine Ligand 12) • PTPRC (Protein Tyrosine Phosphatase Receptor Type C) • EPCAM (Epithelial cell adhesion molecule) • VIM (Vimentin) • FAP (Fibroblast activation protein, alpha) • PVR (PVR Cell Adhesion Molecule) • CD31 (Platelet and endothelial cell adhesion molecule 1) • THY1 (Thy-1 membrane glycoprotein) • MMP1 (Matrix metallopeptidase 1) • PECAM1 (Platelet And Endothelial Cell Adhesion Molecule 1) • S100A4 (S100 calcium binding protein A4)
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CD8 positive • CD44 expression • CXCL12 expression • VIM expression • EPCAM expression
2ms
Utility of circulating tumor DNA (ctDNA) from cerebrospinal fluid (CSF) for prognosis of patients with recurrent high grade glioma (AACR 2024)
Specifically, the presence of enhancing disease and contact of the tumor with the ventricular space were positively associated with detection of CSF ctDNA. Additionally, CSF ctDNA was associated with positive cytology in 21/21 cases (100%).With our improved bioinformatics pipeline we hypothesize ctDNA from CSF may be used as a prognostic biomarker for survival, but confirmation requires further validation in a prospective study.
Clinical • Circulating tumor DNA
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PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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IDH wild-type
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MSK-IMPACT
2ms
Monitoring glioma treatment response through longitudinal analysis of cell-free DNA in cerebrospinal fluid: Insights from a comprehensive study using next-generation sequencing (AACR 2024)
Subsequently, CSF cfDNA decreased during chemoradiation and adjuvant treatment and increased at recurrence, even in patients co-enrolled in immunotherapy trials for IL-7 agonists and pembrolizumab... Analysis of cfDNA throughout a patient's disease course, encompassing resection and treatment with standard-of-care and experimental therapies, may aid in disease monitoring and treatment response. Further evaluation in a larger patient cohort is needed to determine the sensitivity of changes in mutations, CNB, and cfDNA quantity for glioma disease burden.
PD(L)-1 Biomarker • IO biomarker • Next-generation sequencing • Cell-free DNA
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EGFR (Epidermal growth factor receptor) • MAP2K1 (Mitogen-activated protein kinase kinase 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • IL7 (Interleukin 7)
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EGFR amplification
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PredicineCARE™
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Keytruda (pembrolizumab)
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